AimSirolimus, a mammalian target of rapamycin inhibitor, inhibits cell growth and proliferation by controlling ribosome biogenesis and protein synthesis in vascular anomalies and cancers. However, most sirolimus studies on vascular anomalies were conducted in the pediatric population, with limited data in adults. In this study, we assessed the effectiveness and safety of sirolimus in adult patients with vascular malformation, a subtype of vascular anomaly.MethodsWe conducted a retrospective analysis of adult vascular malformation patients aged over 16, treated at Hacettepe University Cancer Institute from January 2013 to September 2022. Patient demographics and clinical characteristics were recorded. The primary outcome was the efficacy of sirolimus evaluated by response and disease control rates. The disease control rate was defined as the cumulative percentage of complete or partial responses, along with stable disease. The secondary endpoint was toxicity and safety.Results38 patients with a median age of 21 (IQR: 18–33) were recruited. Prior to sirolimus treatment, 57.9% of patients had undergone other therapeutic interventions, predominantly sclerotherapy and surgery. The median follow-up time during sirolimus treatment was 18.5 (IQR: 11.3–74.5) months. The disease control rate was 92.1% (35/38). Head-neck localization was associated with better response rates ( p = .001). Sirolimus was generally well tolerated and grade 1 or 2 oral mucositis ( n = 4) and skin rash ( n = 3) were the most common side effects.ConclusionIn this study, we found sirolimus was efficacious and well tolerated in adult patients with vascular malformation.

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西罗莫司治疗成人血管畸形患者的经验

目的西罗莫司是雷帕霉素抑制剂的哺乳动物靶标，通过控制血管异常和癌症中的核糖体生物合成和蛋白质合成来抑制细胞生长和增殖。然而，大多数关于血管异常的西罗莫司研究都是在儿科人群中进行的，成人数据有限。在这项研究中，我们评估了西罗莫司对血管畸形（血管异常的一种亚型）成人患者的有效性和安全性。方法我们对 2013 年 1 月至 9 月在 Hacettepe 大学癌症研究所接受治疗的 16 岁以上成人血管畸形患者进行了回顾性分析2022 年。记录患者人口统计数据和临床特征。主要结局是通过反应和疾病控制率评估西罗莫司的疗效。疾病控制率定义为完全或部分缓解以及疾病稳定的累积百分比。次要终点是毒性和安全性。结果 招募了 38 名中位年龄为 21 岁（IQR：18-33）的患者。在西罗莫司治疗之前，57.9%的患者接受过其他治疗干预，主要是硬化疗法和手术。西罗莫司治疗期间的中位随访时间为 18.5（IQR：11.3-74.5）个月。疾病控制率为92.1%（35/38）。头颈定位与更好的反应率相关 (p = .001)。西罗莫司通常耐受性良好，最常见的副作用是 1 级或 2 级口腔粘膜炎 ( n = 4) 和皮疹 ( n = 3)。结论在这项研究中，我们发现西罗莫司对成人血管畸形患者有效且耐受性良好。